This special event was held within Fabry Disease Awareness Month (April) and is part of the European Innovation Council (EIC) Nano4Rare project, which focuses on developing nanomedicine-based therapies for Fabry disease. Despite uncertainty caused by a blackout the day before, the event was held as planned—thanks to the commitment and flexibility of all speakers and participants.

Guillem PIntos during the Fabry event | ICMAB-CSIC

Building bridges between science and patients

The day opened with welcoming words by Lourdes Fàbrega, Director of ICMAB, and Nora Ventosa, coordinator of the Nano4Rare project (together with Elisabet González), who emphasized the importance of organizing events that foster dialogue between researchers, clinicians, and patients.

Roundtable with Simó Schwartz, Maria Camprodon, Jordi Cruz, Alfredo Romero (patient) moderated by Guillem Pintos | ICMAB-CSIC

Centering the patient experience

One of the central themes of the event was the importance of patient-centered approaches in rare disease research and care. This was strongly emphasized during the roundtable discussions, where speakers discussed the need for early diagnosis, stronger collaborative networks, and patient inclusion in the research process.

A first roundtable on the importance of diagnosis included a Fabry patient, Alfredo Romero, and Simó Schwartz (Vall d'Hebron Hospital), Maria Camprodon (Vall d'Hebron Hospital), Jordi Cruz (MPS Lisosomales and FEDER foundation ) and was moderated by Guillem Pintos (Vall d'Hebron Hospital). A second roundtalbe, in the afteroon, was devoted to R+D challenges on LSD diseases and was moderated by Judit Tomsen (ICMAB-CSIC) and included the participation of Sílvia Muro (IBEC), Míriam Royo (IQAC-CSIC) and Ibane Abasolo (IQAC-CSIC). 

Jordi Cruz, Director of MPS Lisosomales and board member of FEDER, and Francesc Cayuela, President of the Catalan Federation of Rare Diseases (FECAMM), both underlined the vital role of patient organizations in supporting affected individuals and giving visibility to rare conditions. 

Elisabet Gonzàlez presenting the Nano4Rare project at the Fabry event | ICMAB-CSIC 

A look at research and innovation

The program included the presentation of the Nano4Rare project (EIC Transition) by Elisabet Gonzàlez, researcher at the Nanomol-Bio Group (ICMAB-CSIC) and coordinator of the Nano4Rare project, together with Nora Ventosa, as well as the presentation of the NEX-TRY project (ISCIII Instituto de Salud Carlos III) by Ibane Abasolo, leader of the Therapeutic Applications of Nanomedicine Research Group at IQAC-CSIC. These sessions highlighted ongoing efforts to develop innovative therapies for Fabry disease and other rare conditions using advanced nanomedicine approaches. 

Attendees had the opportunity to network and exchange ideas during the coffee break and lunch sessions, which counted with wonderful dishes and was a time to enjoy and reflect. 

The event concluded with a guided visit to Nanomol Technologies, located at the UAB Campus, where participants toured the pilot plant for nanomedicine production, gaining a first-hand look at how research is being translated into therapeutic innovation.

Roundtable with Sílvia Muro, Míriam Royo, Ibane Abasolo moderated by Judit Tomsen | ICMAB-CSIC

Looking ahead

The “Fabry Connections” event was a vivid reminder of the power of collaboration across disciplines. It highlighted how research centers like ICMAB can become vital spaces where patients, scientists, clinicians, and associations come together to inspire progress and co-create a better future for people affected by rare diseases.

We thank all participants, speakers, and supporters for making this event possible and reaffirm our commitment to advancing research, raising awareness, and supporting the rare disease community.

Visit at Nanomol Technologies | ICMAB-CSIC

Take a look here at more photos of the event.

About Nano4Rare

Nano4Rare is a European research project focused on the preclinical development of a nanomedicine candidate for Fabry disease treatment, aiming for clinical application. Nano4Rare is a European Innovation Council (EIC) Transition project, a program aimed for the validation of technologies and the development of business plans for specific applications (TRL 3-6). The project is coordinated by Nora Ventosa and Elisabet González, researchers at the Nanomol-Bio group at ICMAB-CSIC, and is linked to the Phoenix OITB EU project (Pharmaceutical Open Innovation Test Bed for Enabling Nano-pharmaceutical Innovative Products). 

The project is funded by the European Union’s Horizon Europe research and innovation program under grant agreement No. 101136772.

Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or the European Research Executive Agency. Neither the European Union nor the European Research Executive Agency can be held responsible for them.